Experimental Gene Therapy Curbs Progression Of Huntington’s Disease
The results of the small trial have not been published or peer reviewed, but the gene therapy company uniQure is looking to seek approval for the experimental treatment early next year. Plus, news on junk genes, anti-malaria baby wraps, the rollout of cheaper HIV drugs, and more.
An experimental treatment for the first time slowed the devastating progression of Huntington鈥檚 disease, gene therapy company uniQure announced Wednesday, a rare hopeful advance against a cruel genetic disease that robs people of control of their bodies and minds in the prime of life. ... About 40,000 people in the United States have symptomatic Huntington鈥檚, which is caused by a mutated gene. (Johnson, 9/24)
In other innovations 鈥
When couples have trouble conceiving a baby or lose a pregnancy, they often undergo routine tests, which can turn up a shock: One of the prospective parents may be missing a chromosome. The most common chromosomal abnormality 鈥 carried by about 1 in 800 people 鈥 is a 鈥淩obertsonian translocation,鈥 when two chromosomes get fused together. People are often healthy, but one short of the typical 46 chromosomes for a human. Most don鈥檛 learn they carry this genetic anomaly unless they experience reproductive problems and seek testing. (Johnson, 9/24)
Toting around young children in baby wraps treated with insect repellent cut their malaria infection rate by two-thirds, according to findings of a randomized trial in Uganda. Among 400 pairs of moms and children who all used insecticide-treated bed nets at night, the clinical malaria incidence rate for infants fell by 66% for those carried in permethrin-treated wraps rather than sham-treated wraps ... said researchers led by Ross Boyce, MD, of the University of North Carolina at Chapel Hill. (Rudd, 9/24)
On the high costs of prescription drugs 鈥
A twice-yearly injection described as the most promising HIV prevention tool in decades is poised to reach millions more people, with new generic versions priced at about $40 per patient per year. The Gates Foundation and Indian drugmaker Hetero Labs Ltd. are among the groups moving to produce the medication, lenacapavir, which Gilead Sciences Inc. sells in the US for a list price of more than $28,000 annually under the brand name Yeztugo. (Kew and Furlong, 9/24)
Three hours inland from Chennai, India, traffic crawls on a half-finished road past rice fields and cow crossings until it reaches a newer complex of neat white buildings. Among them is the cancer wing of a hospital founded over a century ago by American missionaries. ... A single infusion might cost at least $7,000 at the standard dose in the US, and a year鈥檚 treatment more than $200,000. Here, by financial necessity, most of the patients are getting as little as one-sixth of that. (Langreth, 9/24)
Pharmacy middlemen are working on a proposal to voluntarily change some of their business practices in an effort to avoid new regulation from the Trump administration, according to people familiar with the discussions. The main lobbying group that represents so-called pharmacy benefit managers, the Pharmaceutical Care Management Association, has drafted proposals to bring to the Centers for Medicare and Medicaid Services, according to a document viewed by Bloomberg News. (Tozzi and Cohrs Zhang, 9/24)
University of Maine researchers developed a new process to make HBL, a key ingredient in many medicines, from renewable glucose instead of petroleum. The approach not only lowers drug production costs but also reduces emissions. (University of Maine, 9/21)